October 19, 2017
#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says

As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act ... Read more
October 17, 2017October 17, 2017
New Mutations Account for Genetically Unsolved Cases of EBS, Researchers Say

New mutations in KRT5, the gene coding for keratin 5, one of the major structural components of the skin ... Read more
October 6, 2017October 6, 2017
Abeona to Advance Gene Therapy Research at Commercial Manufacturing Facility in Ohio

Abeona Therapeutics, a biopharmaceutical developing new gene therapies for rare diseases, announced the opening of the first anticipated commercial ... Read more
October 5, 2017October 5, 2017
Austrian Researchers Develop New Formulation of Diacerein to Treat EBS

Diacerein, which is a commercially available medicine used to treat osteoarthritis, also may hold therapeutic potential to treat blistering ... Read more
October 3, 2017October 3, 2017
Fibrocell Reports Positive Results of Phase 1/2 Trial of FCX-007 Gene Therapy for RDEB

Fibrocell Science recently reported positive interim results from its Phase 1/2 clinical trial testing FCX-007 for the treatment ... Read more
September 28, 2017September 28, 2017
Efforts Ramping Up to Treat EB: Interview with DEBRA

Editor’s note: This is the second in a two-part series on epidermolysis bullosa. The first part, about the complications ... Read more
September 26, 2017September 26, 2017
Life with EB: Tough, Time-Consuming, and Fraught with Risk

Editor’s note: First of a two-part series on epidermolysis bullosa. The second part, focusing on treatments ... Read more
September 21, 2017September 21, 2017
FDA Grants Orphan Drug Status to ProQR’s QR-313 as Treatment for Dystrophic EB

The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to ProQR Therapeutics‘ investigational drug QR-313, developed ... Read more
September 19, 2017September 19, 2017
InMed’s Cannabinoid-based Manufacturing System Covered in New Patent Application

InMed Pharmaceuticals has filed a provisional patent application covering its proprietary cannabinoid manufacturing system based on E. coli bacteria. This is ... Read more
September 14, 2017September 14, 2017
Amicus’ SD-101 Cream Misses Goals in Epidermolysis Bullosa Phase 3 Trial

Amicus Therapeutics’ Phase 3 trial of SD-101 cream to treat wounds in patients with epidermolysis bullosa did not meet ... Read more
September 12, 2017September 12, 2017
CRISPR/Cas9 RNP System Shown to Be Effective for Gene Editing in Epidermolysis Bullosa Mice

Researchers have successfully applied the CRISPR/Cas9 system in a mouse model of recessive dystrophic epidermolysis bullosa (RDEB) to restore ... Read more
September 7, 2017September 7, 2017
Low Vitamin D Levels, More Severe Disease Linked to Poor Bone Health in Kids with EB, Study Finds

Low bone mineral density, a measure of bone health, was associated with low vitamin D levels and more severe ... Read more

#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says

As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies ... Read more

October 17, 2017October 17, 2017

New Mutations Account for Genetically Unsolved Cases of EBS, Researchers Say

New mutations in KRT5, the gene coding for keratin 5, one of the major structural components of the skin epidermis, account for previously unsolved cases of basal epidermolysis bullosa ... Read more

October 6, 2017October 6, 2017

Abeona to Advance Gene Therapy Research at Commercial Manufacturing Facility in Ohio

Abeona Therapeutics, a biopharmaceutical developing new gene therapies for rare diseases, announced the opening of the first anticipated commercial gene therapy manufacturing facility in Cleveland, Ohio. The facility is ... Read more

October 5, 2017October 5, 2017

Austrian Researchers Develop New Formulation of Diacerein to Treat EBS

Diacerein, which is a commercially available medicine used to treat osteoarthritis, also may hold therapeutic potential to treat blistering in patients with epidermolysis bullosa simplex type Dowling-Meara (EBS-DM). Initially ... Read more

October 3, 2017October 3, 2017

Fibrocell Reports Positive Results of Phase 1/2 Trial of FCX-007 Gene Therapy for RDEB

Fibrocell Science recently reported positive interim results from its Phase 1/2 clinical trial testing FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). FCX-007 is a gene therapy product candidate, ... Read more

September 28, 2017September 28, 2017

Efforts Ramping Up to Treat EB: Interview with DEBRA

Editor’s note: This is the second in a two-part series on epidermolysis bullosa. The first part, about the complications caregivers experience with the disease, can be found here. Despite ... Read more

September 26, 2017September 26, 2017

Life with EB: Tough, Time-Consuming, and Fraught with Risk

Editor’s note: First of a two-part series on epidermolysis bullosa. The second part, focusing on treatments and care, will follow soon. For caregivers, epidermolysis bullosa (EB) is complicated, time-consuming, ... Read more

September 21, 2017September 21, 2017

FDA Grants Orphan Drug Status to ProQR’s QR-313 as Treatment for Dystrophic EB

The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to ProQR Therapeutics‘ investigational drug QR-313, developed for the treatment of dystrophic epidermolysis bullosa (DEB). A first-in-class ... Read more

September 19, 2017September 19, 2017

InMed’s Cannabinoid-based Manufacturing System Covered in New Patent Application

InMed Pharmaceuticals has filed a provisional patent application covering its proprietary cannabinoid manufacturing system based on E. coli bacteria. This is the first of a series of patent applications the company ... Read more

September 14, 2017September 14, 2017

Amicus’ SD-101 Cream Misses Goals in Epidermolysis Bullosa Phase 3 Trial

Amicus Therapeutics’ Phase 3 trial of SD-101 cream to treat wounds in patients with epidermolysis bullosa did not meet the company’s primary and secondary treatment goals, Amicus announced. The ... Read more